{"id":204904,"date":"2025-10-06T11:41:07","date_gmt":"2025-10-06T11:41:07","guid":{"rendered":"https:\/\/www.newsbeep.com\/us\/204904\/"},"modified":"2025-10-06T11:41:07","modified_gmt":"2025-10-06T11:41:07","slug":"opus-genetics-reports-positive-pediatric-data-from-lca5-gene-therapy-study","status":"publish","type":"post","link":"https:\/\/www.newsbeep.com\/us\/204904\/","title":{"rendered":"Opus Genetics reports positive pediatric data from LCA5 gene therapy study"},"content":{"rendered":"

Opus Genetics, Inc. released positive data<\/a> from a pediatric cohort in an ongoing phase 1\/2 clinical study evaluating OPGx-LCA5, an investigational gene therapy, for the treatment of Leber congenital amaurosis type 5 (LCA5).<\/p>\n

This reporting comes just weeks after the company dosed its first patient<\/a> in a separate phase 3 study for phentolamine 0.75%\u2014and received FDA clearance<\/a> for the investigational new drug (IND) of yet another candidate (OPGx-BEST1).<\/p>\n

Opus has certainly been busy \u2026 but let\u2019s begin with LCA5.<\/p>\n

LCA is an inherited retinal disease caused by photoreceptor degeneration and\/or dysfunction.<\/p>\n

More specifically, LCA5 develops as a result of mutations in the LCA5 gene (known to encode lebercilin, a key protein for photoreceptor structure and function.<\/p>\n

Out of all LCA cases, LCA5 reportedly accounts for around 2%.<\/p>\n

And more importantly: There\u2019s currently no treatment approved for LCA5-linked inherited retinal degeneration.<\/p>\n

Which brings us to this LCA5 therapeutic.<\/p>\n

Indeed. OPGx-LAC5 relies on an adeno-associated virus (AAV) vector to deliver a functional LCA5 gene (which encodes a protein essential for photoreceptor function) to photoreceptors within the retina that, in turn, target LCA5.<\/p>\n

Its mode of delivery is via a single, unilateral subretinal injection.<\/p>\n

And on the regulatory side: The FDA granted Rare Pediatric Disease designation<\/a> in August 2024.<\/p>\n

Moving on to \u2026 clinical data.<\/p>\n

OPGx-LCA5 was initially studied in the preclinical stage, in which LCA5-based animal and human induced pluripotent stem cells<\/a> (iPSCS) demonstrated visual function preservation when the candidate was administered before LCA5 reached its highest severity.<\/p>\n

And more recently?<\/p>\n

This is the ongoing 12-month clinical trial we mentioned earlier: An open-label and dose-escalation phase 1\/2 study (NCT05616793<\/a>) evaluating OPGx-LCA5 (in three doses) among 15 adults and pediatric patients (aged 13+) with the LCA5 gene.<\/p>\n

See here<\/a> for participant details, the study setup, and outcome measures.So results have already been reported from this?<\/p>\n

Indeed\u2014preliminary 1-month findings<\/a>\u2014in April 2025, based on one pediatric patient receiving a single subretinal injection of OPGx-LCA5.<\/p>\n

Check out that data<\/a>, as well as early clinical proof-of-concept data<\/a> (reported before that) from the first three adult patients dosed after 6 months.<\/p>\n

Got it. Now to these new findings.<\/p>\n

The latest reporting on the study\u2019s pediatric cohort is based on 3-month data from three patients aged 16 to 17 with severe baseline vision impairment.<\/p>\n

Following a single subretinal injection of OPGx-LCA5, improvements were noted in four key measures of the patients\u2019 visual function.<\/p>\n

\u2026 I\u2019m listening \u2026Visual acuity (VA): A group average of 0.3 logMAR improvement\u2014which was greater than that observed in the adult cohort.Full-field stimulus testing: All patients demonstrated improvements<\/a> in their treated eyes from 1 month and showed >1 log unit improvement in cone sensitivity to both red and blue light.Notably, \u201cthese changes provide evidence of recovery in retinal sensitivity,” Opus stated<\/a>.Multi-luminance orientation and mobility test (MLoMT): All three patients identified more objects<\/a> through the 3-month mark versus baseline, with two out of three having greater improvement in the treated eye versus the control eye.Microperimetry: At screening, two-thirds of patients could not conduct a microperimetry test due to poor VA and nystagmus; microperimetry data was collected from one patient\u2014who demonstrated \u201cearly fixation stability<\/a> \u2026 consistent with functional retinal recovery.\u201dNice! So what does this mean for VA across all studied participants thus far?<\/p>\n

Positive results across the board.<\/p>\n

Opus also reported<\/a> that combined adult data from the study indicated VA improvements were sustained through 18 months\u2014\u201cboth in terms of mean change from baseline and mean interocular difference, underscoring the potential durability of the treatment response.\u201d<\/p>\n

Any adverse events to report?<\/p>\n

In all six patients (three adults and pediatric patients each) dosed to date, OPGx-LCA5 has been \u201cwell-tolerated.\u201d<\/p>\n

There have also been no ocular serious adverse events (AEs) or dose-limiting toxicities observed, and all reported<\/a> ocular AEs that have been anticipated and \u201cmild in severity\u201d\u2014with none related to the OPGx-LCA5 itself.<\/p>\n

So what\u2019s the significance of these findings?<\/p>\n

While the study is still ongoing (expected to conclude in 2028, according to ClinicalTrials.gov<\/a>), principal investigator Tomas S. Aleman, MD, noted<\/a> that the data thus far support \u201cgene augmentation therapy can potentially restore cone function in patients with LCA5.\u201d<\/p>\n

And what\u2019s next?<\/p>\n

Next up, Opus expects to meet with the FDA sometime in Q4 2025 (so between now and Dec. 31).<\/p>\n

We\u2019ll keep you up to date on the latest!<\/p>\n","protected":false},"excerpt":{"rendered":"Opus Genetics, Inc. released positive data from a pediatric cohort in an ongoing phase 1\/2 clinical study evaluating…\n","protected":false},"author":2,"featured_media":204905,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[50],"tags":[200,79],"class_list":["post-204904","post","type-post","status-publish","format-standard","has-post-thumbnail","category-genetics","tag-genetics","tag-science"],"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts\/204904","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/comments?post=204904"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts\/204904\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/media\/204905"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/media?parent=204904"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/categories?post=204904"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/tags?post=204904"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}